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RecruitingLast updated: 29 November 2023

This study is investigating targeted therapy (bezuclastinib) for the treatment of advanced systemic mastocytosis (AdvSM), including people with Aggressive SM( ASM), SM with associated haematologic neoplasm (SM-AHN), and Mast Cell Leukaemia (MCL)A Phase 2 Open-Label, Multicenter Clinical Study of the Safety, Efficacy, Pharmacokinetic, and Pharmacodynamic Profiles of CGT9486 as a Single Agent in Patients With Advanced Systemic Mastocytosis

Clinical summary


There are two parts to this study, including Part I (Dose Optimisation), and Part II Expansion. Part II will be separated into two stages. Participants will receive bezuclastinib, which will be given as tablets to be taken orally, continuously in 28-day cycles.


This trial is treating patients with mast cell leukaemia, aggressive systemic mastocytosis, and systemic mastocytosis with an associated haematologic neoplasm


Blood Cancers Haematological





More information

Trial Identifiers

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Trial sponsor

Cogent Biosciences, Inc

Scientific Title

A Phase 2 Open-Label, Multicenter Clinical Study of the Safety, Efficacy, Pharmacokinetic, and Pharmacodynamic Profiles of CGT9486 as a Single Agent in Patients With Advanced Systemic Mastocytosis



  1. Diagnosed with 1 of the following advanced mastocytosis diagnoses by Eligibility Committee

    1. Aggressive Systemic Mastocytosis (ASM)
    2. Systemic Mastocytosis with an Associated Hematologic Neoplasm (SM-AHN)
    3. Mast Cell Leukemia (MCL)
  2. Measurable disease according to modified IWG-MRT-ECNM criteria. (A subset of patients inevaluble per mIWG-MRT-ECNM will be included in the study).
  3. ECOG (0 to 3)
  4. Have clinically acceptable local laboratory screening results (clinical chemistry, hematology) within certain limits.


  1. Persistent toxicity from previous therapy for Advanced Systemic Mastocytosis that has not resolved to ≤ Grade 1
  2. Associated hematologic neoplasm requiring immediate antineoplastic therapy
  3. Clinically significant cardiac disease
  4. Known positivity for the FIP1L1 PDGFRA fusion. Patients with eosinophilia without detectable KIT D816V mutation must also lack the PDGFRA fusion mutation prior to enrollment
  5. Seropositive for human immunodeficiency virus (HIV) 1 or 2, or positive for hepatitis B surface antigen or hepatitis C virus (HCV) antibody
  6. History of clinically significant bleeding event within 30 days before the first dose of study drug or need for therapeutic anticoagulation on study
  7. Diagnosed with or treated for malignancy other than the disease under study within the prior 3 years before enrollment
  8. Received any cytoreductive therapy or any investigational agent less than 14 days, and for cladribine, interferon alpha, pegylated interferon, and any antibody therapy less than 28 days, before screening bone marrow biopsy
  9. Received hematopoietic growth factor support within 14 days before the first dose of study drug
  10. Received strong CYP3A4 inhibitors or inducers within 14 days or 5 drug half-lives, whichever is longer, before the first dose of study drug.
  11. Need for treatment with high dose steroids.


  • You are able to swallow medication by mouth.


  • You have been diagnosed with a prior or secondary type of cancer.
  • You have certain types of non-cancer medical conditions.
  • You have had certain treatments, surgical procedures or drugs.

Clinical trials have complex eligibility criteria.

Ask your doctor if this trial could be right for you.

Participating hospitals

This trial is operating as a teletrial at one or more locations. Learn more about teletrials.

Recruiting hospitals

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